Boston To doctors' amazement, experimental new medicines are rescuing people from the brink of blindness so they can read and drive and sometimes even regain perfect vision. These lucky few are the first beneficiaries of an entirely new category of drugs that many hope will revolutionize the care of common eye diseases.
Several competing medicines are in development, all based on similar principles. They are designed to stop the two top causes of adult blindness the "wet" form of macular degeneration, which affects the elderly, and diabetic retinopathy, the biggest source of blindness in working-age people.
Vision loss seems halted for most if they take the drugs soon after their symptoms begin. Some experience stunning reversals of what would have been inevitable blindness.
"I'm telling you, it's miraculous," Eileen Russell said.
Russell, 76, of Worcester, lost vision in her right eye four years ago. In May, her left eye went bad, too, and she was declared legally blind.
But after four injections of one of the drugs, her left eye is 20-25. She drives and reads and is thinking about returning to work as a nurse.
"Yesterday, I had to write a check," she said. "It looked beautiful, right on the line, with a regular pen. I can do all the little things again.''
Around the country, about 70 patients with wet macular degeneration have been treated with the same drug as Russell, Genentech's rhuFab. About half were treated by Dr. Jeffrey Heier of Ophthalmic Consultants of Boston, who said, "I can honestly say I have never seen anything as exciting as this.''
Experts caution that most of the results from the studies on this and similar drugs will not be known for at least a year or two. And for now, the treatments are available only to study volunteers.
None of the drugs are intended for the more common but less aggressive "dry" kind of macular degeneration, nor will they work after eyesight has been gone for months.
Guessing the drugs' ultimate effectiveness based on early testing is risky. Still, doctors estimate that roughly one-quarter to one-third of people with newly diagnosed wet macular degeneration have had significant improvement in their eyesight. In most of the rest, loss of sight is stopped, at least temporarily.
Among others helped by rhuFab is Ernest Hayeck, a retired judge in Worcester, 40 miles west of Boston. One day last September, he discovered he was quickly going blind in his right eye. Doorways looked wavy, and everything was dim.
Doctors said they could do nothing for him. With wet macular degeneration, vision in that eye would cloud to little or nothing within a few months at best. Hayeck was an active retiree, nine years off the state Superior Court but busy on the faculty of the National Judicial College and the board of Wendy's International.
"I was resigned to it," he said. "I told myself I had had 77 good years."
Like a miracle
But when told of Heier's rhuFab study, he seized the chance, even though it meant getting shots in his bad eye. In October, the judge got his first, which he said was painless. By then his sight had failed to 20-100.
"I have achieved what I consider to be a miraculous result," Hayeck said. "My eyesight came back with a vengeance. By the time I had the fourth treatment, I was 20-20 with my glasses on.''
Another of Heier's patients, Edward Nowak, 81, an outdoor writer and photographer in suburban Needham, found vision in his left eye improved from 20-400 last November to 20-50 now.
"The results have been miraculous," he said. "You would think the good Lord himself did this."
Vision restoration possible
Dr. Steven Schwartz, chief of the retina division at UCLA's Jules Stein Eye Institute, has worked with several of the new drugs. "For the first time in my career, I have actually been able to restore vision in patients who otherwise would never be able to get back their central vision," he says. "It is a spectacular advance."
His macular degeneration patients include the actor Dabney Coleman, who in a week on rhuFab went from 20-400 to 20-40 in his left eye and returned to playing tennis.
Increase in cases
An estimated 200,000 new cases of wet macular degeneration are diagnosed in the United States annually. About 4 million U.S. diabetics have some degree of retinopathy, and 24,000 go blind each year.
Both diseases result from misguided growth of blood vessels in the eyes. Since the new drugs attack this underlying problem, doctors hope they will work for both diseases.
The need for new treatments is especially dire in wet macular degeneration, because nothing can be done for most victims. Blindness often follows within months or even weeks of the first symptoms.
It occurs when leaky blood vessels sprout behind the retina, probably in a mistaken attempt to fix the slow breakdown of light-sensitive cells that occurs with age.
These vessels ooze fluid and damage the fragile tissue that controls straight-ahead vision.
The new drugs vary, although most of them, like rhuFab, zero in on a growth-promoting protein called vascular epidermal growth factor, or VEGF. It appears to be an especially important trigger of damaging blood vessels in both forms of blindness.
Alternatives are out there
Other drugs in testing include:
l Anecortave acetate from Alcon, a new steroid injected next to the eye once every six months for macular degeneration.
l Eyetech Pharmaceuticals' EYE001, which is injected into the eyeball like rhuFab for macular degeneration.
l Bausch & Lomb's Retisert implant, which exudes a steroid into the eye for up to three years and is being used for diabetic retinopathy and macular degeneration.
l Lilly's LY333531, the only pill among the new drugs; used to prevent worsening eye disease in diabetics.
Development of these drugs is gratifying to Dr. Judah Folkman of Boston's Children's Hospital, whose three decades of pioneering research into blood vessels provided their scientific basis. Folkman's goal is a cancer treatment, since new blood vessels are necessary for tumor growth.
"Sometimes the most exciting thing in a scientist's career,'' he says, "is an unexpected outcome from one's work."
Nevertheless, experts caution that until the big studies are finished, no one can be sure how well the drugs will work. No one knows how long patients will need to take them, how often disease will return or whether the repeated eye injections have any hazards.
"The early data are very exciting, but it would be premature to extrapolate to cures or use other such adjectives to describe these isolated but impressive vision recoveries," said Dr. Karl Csaky of the National Eye Institute.
"Even if these drugs are as successful as the stockholders' wildest dreams, we'll still need something better," said Dr. David Weissgold of the University of Vermont, "because they won't make the problems go away."